Ocugen’s Geographic Atrophy and Stargardt Gene Therapies Garner Positive ATMP Opinions from EMA
According to Ocugen, the EMA has granted OCU410 and OCU410ST Advanced Therapy Medicinal Product classification.
This article originally appeared on our sister site, Ophthalmology Times Europe®.
The European Medicines Agency (EMA) and its Committee for Advanced Therapies (CAT) has provided a positive opinion to 2 of Ocugen's investigational gene therapy products: OCU410 and OCU410ST.1 OCU410 is under evaluation for the treatment of vision loss due to geographic atrophy (GA) associated with dry age-related macular degeneration (AMD) and OCU410ST is intended to treat Stargardt disease, which is associated with ABCA4-related retinopathies.
According to Ocugen, the EMA has granted OCU410 and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification. The designation allows for an accelerated timeline for regulatory review, in addition to scientific guidance and protocol assistance provided by the EMA.
Notably, the move comes just a month after Ocugen's OCU400, a gene therapy intended to treat retinitis pigmentosa, received ATMP designation from the agency in early February.2
OCU410 and OCU410ST both utilise an adeno-associated virus vector platform for gene delivery. Preliminary 9-month data assessing OCU410 showed considerably lower lesion growth and clinically meaningful visual function gains for patients with GA. The 6-month data from phase 1 of the OCU410ST GARDian clinical trial demonstrated slower lesion growth from baseline in treated eyes versus untreated fellow eyes, as well as clinically meaningful visual improvement in patients with Stargardt disease.1
“The novel modifier gene in OCU410 and OCU410ST targets all 4 pathways linked with dAMD and Stargardt and is delivered through a single subretinal injection as a one-and-done treatment," Huma Qamar, MD, MPH, CMI, the chief medical officer of Ocugen, said in a statement.1 We are very pleased with the structural and functional outcomes demonstrated by both of these candidates, along with a stellar safety profile.”
Shankar Musunuri, PhD, MBA, chairman, CEO and co-founder of Ocugen, added that the ATMP designation could help OCU410 and OCU410ST address patients’ unmet needs in the very near future. “Dosing of phase 2 in the ongoing OCU410 ArMaDa clinical trial is complete, and we are on track to initiate the phase 3 clinical trial next year to pursue potential Marketing Authorisation Application and biologics license application filings in 2028,” he said.1 “Last week, the US Food and Drug Administration endorsed Ocugen’s plan to move forward with a phase 2/3 pivotal confirmatory clinical trial for OCU410ST, which can be the basis of BLA and potential MAA submissions in 2027.”
